HUE029556T2 - Reduced immunoreactivity of AAV virions and their use - Google Patents
Reduced immunoreactivity of AAV virions and their use Download PDFInfo
- Publication number
- HUE029556T2 HUE029556T2 HUE04755800A HUE04755800A HUE029556T2 HU E029556 T2 HUE029556 T2 HU E029556T2 HU E04755800 A HUE04755800 A HU E04755800A HU E04755800 A HUE04755800 A HU E04755800A HU E029556 T2 HUE029556 T2 HU E029556T2
- Authority
- HU
- Hungary
- Prior art keywords
- aav
- amely amely
- virion
- dna
- capsid
- Prior art date
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/482—Serine endopeptidases (3.4.21)
- A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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- A—HUMAN NECESSITIES
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- A—HUMAN NECESSITIES
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- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14161—Methods of inactivation or attenuation
- C12N2750/14162—Methods of inactivation or attenuation by genetic engineering
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Diabetes (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Immunology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Obesity (AREA)
- Neurology (AREA)
- Cardiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Physical Education & Sports Medicine (AREA)
- Emergency Medicine (AREA)
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Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US48039503P | 2003-06-19 | 2003-06-19 | |
US56731004P | 2004-04-30 | 2004-04-30 | |
US57650104P | 2004-06-03 | 2004-06-03 |
Publications (1)
Publication Number | Publication Date |
---|---|
HUE029556T2 true HUE029556T2 (en) | 2017-03-28 |
Family
ID=33545345
Family Applications (5)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HUE13163182A HUE034597T2 (en) | 2003-06-19 | 2004-06-21 | Reduced immunoreactivity of AAV virions and their applications |
HUE11158742A HUE034546T2 (en) | 2003-06-19 | 2004-06-21 | Reduced immunoreactivity of AAV virions and their applications |
HUE17162137A HUE054046T2 (hu) | 2003-06-19 | 2004-06-21 | Csökkentett immunreaktivitású AAV virionok és alkalmazásaik |
HUE04755800A HUE029556T2 (en) | 2003-06-19 | 2004-06-21 | Reduced immunoreactivity of AAV virions and their use |
HUE13163168A HUE034785T2 (en) | 2003-06-19 | 2004-06-21 | Reduced immunoreactivity of AAV virions and their use |
Family Applications Before (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HUE13163182A HUE034597T2 (en) | 2003-06-19 | 2004-06-21 | Reduced immunoreactivity of AAV virions and their applications |
HUE11158742A HUE034546T2 (en) | 2003-06-19 | 2004-06-21 | Reduced immunoreactivity of AAV virions and their applications |
HUE17162137A HUE054046T2 (hu) | 2003-06-19 | 2004-06-21 | Csökkentett immunreaktivitású AAV virionok és alkalmazásaik |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HUE13163168A HUE034785T2 (en) | 2003-06-19 | 2004-06-21 | Reduced immunoreactivity of AAV virions and their use |
Country Status (11)
Country | Link |
---|---|
US (6) | US7259151B2 (pt) |
EP (5) | EP2357189B1 (pt) |
JP (7) | JP2007524386A (pt) |
CY (5) | CY1117575T1 (pt) |
DK (5) | DK2357189T3 (pt) |
ES (5) | ES2627913T3 (pt) |
HU (5) | HUE034597T2 (pt) |
PL (5) | PL3235827T3 (pt) |
PT (5) | PT1633772E (pt) |
SI (5) | SI2657248T1 (pt) |
WO (1) | WO2004112727A2 (pt) |
Families Citing this family (164)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20030215422A1 (en) | 1996-09-11 | 2003-11-20 | John A. Chiorini | Aav4 vector and uses thereof |
WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
WO2000075365A2 (en) | 1999-06-08 | 2000-12-14 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
CN102181480B (zh) | 2001-11-13 | 2016-01-27 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
US7419817B2 (en) | 2002-05-17 | 2008-09-02 | The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. | Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography |
EP1608763A2 (en) | 2003-03-31 | 2005-12-28 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
WO2005017101A2 (en) | 2003-05-19 | 2005-02-24 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH | Avian adenoassociated virus (aaav) and uses thereof |
DK2357189T3 (en) | 2003-06-19 | 2017-06-19 | Genzyme Corp | AAV virions with reduced immunoreactivity and applications thereof |
US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
US9233131B2 (en) * | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
US8137960B2 (en) | 2003-12-04 | 2012-03-20 | The United States Of America As Represented By The Department Of Health And Human Services | Bovine adeno-associated viral (BAAV) vector and uses thereof |
US20080188431A1 (en) * | 2004-09-08 | 2008-08-07 | Chiorini John A | Transcytosis of Adeno-Associated Viruses |
EP1828390B1 (en) * | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
CN102994549B (zh) * | 2005-04-07 | 2015-02-11 | 宾夕法尼亚大学托管会 | 增强腺相关病毒载体功能的方法 |
US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
JP2009535034A (ja) * | 2006-04-28 | 2009-10-01 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | 哺乳動物細胞のパルボウイルス形質導入を調節若しくはウイルス感染を変えるための方法および化合物、ウイルス受容体若しくは共受容体の同定方法 |
EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
ES2429142T3 (es) | 2009-01-30 | 2013-11-13 | Uniqure Ip B.V. | Agentes terapéuticos de alanina-glioxilato aminotransferasa |
ES2634118T3 (es) | 2009-02-11 | 2017-09-26 | The University Of North Carolina At Chapel Hill | Vectores de virus modificados y métodos para fabricar y utilizar los mismos |
CA2756596A1 (en) | 2009-03-27 | 2010-09-30 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
EP2287323A1 (en) | 2009-07-31 | 2011-02-23 | Association Institut de Myologie | Widespread gene delivery to the retina using systemic administration of AAV vectors |
WO2011032100A1 (en) | 2009-09-11 | 2011-03-17 | Government Of The U.S.A., As Represented By The Secretary, Department Of Health And Human Services | Inhibitors of kshv vil6 and human il6 |
MX2012005340A (es) | 2009-11-05 | 2012-12-05 | Proyecto Biomedicina Cima Sl | Sistemas de expresion regulada. |
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US20060292117A1 (en) * | 2002-04-17 | 2006-12-28 | Loiler Scott A | Improved rAAv vectors |
DK2357189T3 (en) | 2003-06-19 | 2017-06-19 | Genzyme Corp | AAV virions with reduced immunoreactivity and applications thereof |
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